A Phase 1/2, Open Label, Dose Ranging Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Exploratory Efficacy Parameters of PRX-102 Administered by Intravenous Infusion Every 2 Weeks for 12 Weeks to Adult Fabry Patients

Protalix Ltd.0 个研究点目标入组 18 人开始时间: 2013年9月5日最近更新: 2015年8月4日

适应症

试验速览

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•1.Symptomatic adult Fabry patients (> or = to 18 yrs, males and females)
•2.Males: plasma and/or leucocyte alpha galactosidase activity (by activity assay) less than lower limit of normal (LLN in plasma=3.2 nmol/hr/ml, LLN in leucocytes=32 nmol/hr/mg/protein)
•3.Females: historical genetic test results consistent with Fabry mutations
•4.Globotriaosylceramide (Gb3) concentration in urine > 1.5 times upper normal limit
•5.Patients who have never received enzyme replacement therapy (ERT) in the past, or patients who have not received ERT in the past 6 months and have a negative anti PRX-102 antibody test
•6.Chronic kidney disease - stages 1 or 2 (CKD1 or 2) (Appendix 7) with proteinuria > 200 mg/g protein-to-creatinine ratio measured in a Spot urine sample or equivalent demonstrated in at least one of 2 separate samples (one sample in screening visit and the other from historical data)
•7.The patient signs informed consent
•8.Female patients and male patients whose co-partners are of child-bearing potential agree to use a medically acceptable method of contraception, not including the rhythm method
•Are the trial subjects under 18? no
•Number of subjects for this age range:
•F.1.2 Adults (18-64 years) yes
•F.1.2.1 Number of subjects for this age range 17
•F.1.3 Elderly (>=65 years) yes
•F.1.3.1 Number of subjects for this age range 1

•1.Participation in any trial of an investigational drug within 30 days prior to study screening
•2.Chronic kidney disease stages 3-5 (CKD 3-5) (Appendix 7)
•3.History of dialysis or renal transplantation
•4.Angiotensin converting enzyme (ACE) inhibitor or angiotensin receptor blocker (ARB) therapy initiated or dose changed in the 4 weeks prior to screening
•5.Severe myocardial fibrosis by MRI (> or = to 2 late-enhancement [LE] positive left ventricular segments) (Weidemann et al. 2009)
•6.History of clinical stroke
•7.Pregnant or nursing
•8.Presence of HIV and/or HBsAg and/or Hepatitis C infections
•9.Known allergies to ERT
•10.Known allergy to Gadolinium based contrast agents
•11.Presence of any medical, emotional, behavioral or psychological condition that, in the judgment of the Investigator and/or Medical Director, would interfere with the patients compliance with the requirements of the study

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